Our history is closely tied to the C3P3 technology – the first ever non-viral artificial eukaryotic expression system developed from scratch by Eukarÿs.
C3P3 is the world’s first non-viral, artificial eukaryotic expression system that autonomously synthesizes the messenger RNA (s) of interest (mRNA) in the cytoplasm of the host cell in a high-throughput and efficient manner, allowing high expression of the target proteins.
Until late 2000s, the development of an artificial eukaryotic expression system was nearly impossible due to the amazing complexity of eukaryotic post-transcriptional mRNA modifications.
The emergence of synthetic biology during the last decade was pivotal, allowing the construction of the C3P3 expression system (stands for chimeric cytoplasmic capping prone phage polymerase).
The development of the first generation of C3P3 (C3P3-G1) was completed by the end of 2016 with good performances with CpG-free DNA templates, which are preferably used for our synthetic gene therapy – a novel therapeutic approach designed as safe, efficient and operating for most human disorders.
Eukarÿs is currently finalizing the third generation of its C3P3 system (C3P3-G3b) with much greater complexity and performances than the initial prototype. C3P3-G3b is used for both synthetic gene therapy and bioproduction tools.
Today, Eukarÿs is a preclinical-stage biotechnology company that develops a pipeline of synthetic gene therapy treatments for liver diseases. Synthetic gene therapy is a radically novel therapeutic approach, which exploits the C3P3 system and is designed to be safe and efficient for the treatment of either rare monogenic diseases or frequent multifactorial complex disorders. The advanced prototype of EUK-LPR, our first synthetic gene therapy treatment, has demonstrated excellent performances in animal models. Further therapeutic programs will be launched in 2019.
Tomorrow, Eukarÿs will extend its synthetic gene therapy approach to other therapeutic areas taking advantage of its generic nature. The therapeutic areas to be addressed will be diseases associated with secreted protein deficiency and the field of immuno-oncology.
Besides its therapeutic development, Eukarÿs develops tools for the bioproduction of viruses and proteins, which also exploit the C3P3 artificial expression system. These tools have already shown excellent performances for the bioproduction of viruses and proteins. These tools will be available for research purposes.